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New study tracks long-term safety of DMD drug vamorolone in boys

NCT ID NCT06564974

Disease control Sponsor: Catalyst Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 21, 2026 · Updated 2 times

Summary

This study follows 250 boys (ages 2 and older) with Duchenne muscular dystrophy who are taking vamorolone (AGAMREE). Researchers will monitor side effects, growth, bone health, heart function, and quality of life over time. The goal is to better understand the long-term safety and real-world benefits of this treatment.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 250 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2024

Expected to finish

Feb 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Male patients at least 2 years old with confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti-dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).

Ages

2 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures. 2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires. 3. Male patients at least 2 years old. 4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot). 5. Patient has a current, active prescription for, or is on, AGAMREE®. Exclusion Criteria: 1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.

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Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Duchenne muscular dystrophy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    33 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Arkansas Childrens Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Atrium Health Neurosciences Institute

    RECRUITING

    Charlotte, North Carolina, 28207, United States

  • Children's Hospital Los Angeles

    NOT_YET_RECRUITING

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia (CHOP)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National Health System

    NOT_YET_RECRUITING

    Washington D.C., District of Columbia, 20010, United States

  • Children's Wisconsin

    NOT_YET_RECRUITING

    Milwaukee, Wisconsin, 53226, United States

  • Cincinnati Children's Hospital Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center and Childrens Health Center

    RECRUITING

    Durham, North Carolina, 27710, United States

  • FDI Clinical Research

    RECRUITING

    San Juan, Puerto Rico, 00927, Puerto Rico

  • Helen DeVos Children's Hospital

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Indiana University Health - Riley Hospital for Children

    RECRUITING

    Indianapolis, Indiana, 46202, United States

  • Johns Hopkins All Children's Hospital

    NOT_YET_RECRUITING

    St. Petersburg, Florida, 33701, United States

  • Loma Linda University Pediatric Subspecialty Clinics

    NOT_YET_RECRUITING

    Loma Linda, California, 92354, United States

  • Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Masonic Children's Hospital - Discovery Clinic

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • Nemours Children's Hospital

    RECRUITING

    Orlando, Florida, 32827, United States

  • Neurology Rare Disease Center - Neurology & Neuromuscular Care Center

    RECRUITING

    Denton, Texas, 76208, United States

    Contact Email: •••••@•••••

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

  • Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute

    RECRUITING

    Hershey, Pennsylvania, 17033, United States

  • Phoenix Children's Hospital

    RECRUITING

    Phoenix, Arizona, 85016, United States

  • San Jorge Children's Hospital

    RECRUITING

    San Juan, Puerto Rico, 00912, Puerto Rico

  • Seattle Children's Hospital

    RECRUITING

    Seattle, Washington, 98105, United States

  • Stanford University

    NOT_YET_RECRUITING

    Palo Alto, California, 94305, United States

  • The Children's Hospital at Montefiore

    NOT_YET_RECRUITING

    The Bronx, New York, 10467-2403, United States

  • The University of Texas Health Science Center at San Antonio

    RECRUITING

    San Antonio, Texas, 78229, United States

  • University of California, Davis

    NOT_YET_RECRUITING

    Sacramento, California, 95817, United States

  • University of Florida Clinical and Translational Science Institue

    NOT_YET_RECRUITING

    Gainesville, Florida, 32610, United States

  • University of Kansas Medical Center

    RECRUITING

    Kansas City, Kansas, 66205, United States

  • University of Massachusetts Memorial Medical Center

    RECRUITING

    North Worcester, Massachusetts, 01655, United States

  • University of Miami

    NOT_YET_RECRUITING

    Miami, Florida, 33136, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75235, United States

  • University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • Valley Children's Hospital

    NOT_YET_RECRUITING

    Madera, California, 93636, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.